NCT06131983

Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1

A Phase1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 16 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Conditions

• Facio-Scapulo-Humeral Dystrophy

Interventions

Type	Name	Description
DRUG	ARO-DUX4 for Injection	single or multiple doses of ARO-DUX4 by intravenous (IV) infusion
DRUG	Placebo	calculated volume to match active treatment by IV infusion

Timeline

• Start date: 2024-02-22
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2023-11-15
• Last updated: 2026-02-06

Locations

17 sites across 8 countries: Australia, Canada, Germany, Italy, Netherlands, New Zealand, Spain, Thailand

Regulatory

• FDA-regulated drug study